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The data also provided enough indications of efficacy — by sending "healthies" to sleep — to allow Merck to accelerate its process, and skip a formal proof-of-concept stage.
Results emerging from clinical trials with oncolytic viruses demonstrate the safety and feasibility of a virotherapeutic approach and provide early indications of efficacy.
Despite the indications of efficacy, studies (particularly of farmers and applicators) show that PPE is frequently not used (e.g., Perry et al. 2002).
This study suggested that a randomised controlled trial of counselling in schools is acceptable and feasible, although initial indications of efficacy are mixed.
Since then, many clinical studies have been published with largely positive indications of efficacy (Table 2) (review articles [ 54, 87, 103– 103]).
Although these approaches have demonstrated tantalising indications of efficacy in a small group of individuals, they have not reached the threshold of therapeutic efficacy or safety.
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These are then pooled in a meta-analysis to give an overall indication of efficacy.
Others are pilot studies that should give an indication of efficacy to encourage the initiation of a fully powered trial.
These findings strongly suggest a potential value in employing such composite endpoints in future prospective small studies designed to establish an early indication of efficacy.
Surrogate markers for relapse are therefore required to obtain a preliminary indication of efficacy and sterilising activity of the drug, even though a phase III trial with estimation of relapse rates is eventually necessary.
Therefore, surrogate endpoints that can be measured sooner and more frequently during the course of a clinical trial, are being used to provide an earlier indication of efficacy [ 9].
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Justyna Jupowicz-Kozak
CEO of Professional Science Editing for Scientists @ prosciediting.com